Expert review of pharmacoeconomics & outcomes research · 2026
Defining value criteria for outcomes-based managed entry agreements in CAR-T therapy for relapsed/refractory multiple myeloma: a multi-criteria decision analysis study.
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Mateos Manteca María Victoria, Poveda Andrés José Luis, Oriol Rocafiguera Albert, Escudero-Vilaplana Vicente, Trillo Mata José Luis, Gómez Pajuelo Pedro, Arribas Ruiz Alberto, Domínguez Lubillo Teresa, Vidal-Vilar Neus, Ariznavarreta Martín Asís
Аннотация
BACKGROUND: This study aimed to identify and prioritize criteria for designing and monitoring outcomes-based managed entry agreements (MEAs) for chimeric antigen receptor T-cell (CAR-T) therapy in relapsed/refractory multiple myeloma (RRMM), from the perspective of healthcare professionals and decision-makers.
RESEARCH DESIGN AND METHODS: A structured MCDA-informed expert weighting exercise was conducted using an adapted EVIDEM framework. The study comprised two phases: a) selection and structuring of criteria through a targeted literature review and scientific committee (SC) consensus; and b) nonhierarchical weighting of criteria and sub-criteria by a multidisciplinary expert group (n = 19) of hospital pharmacists, physicians, and evaluators/decision-makers.
RESULTS: Twelve criteria were identified. Following SC discussion, five criteria were selected for weighting: efficacy, safety, patient-reported outcomes, treatment cost, and other direct medical costs. Among the weighted sub-criteria, overall survival (OS) and progression-free survival (PFS) achieved the highest scores, with mean values of 4.21 (95% CI: 3.68-4.74) and 4.05 (95% CI: 3.52-4.58), respectively.
CONCLUSIONS: In this structured MCDA-informed expert weighting exercise, OS and PFS were identified as the highest-priority value criteria for designing and monitoring outcomes-based MEAs for CAR-T therapies in RRMM. These findings may support feasible, evidence-based reimbursement models but require validation in larger and more diverse stakeholder groups.
RESEARCH DESIGN AND METHODS: A structured MCDA-informed expert weighting exercise was conducted using an adapted EVIDEM framework. The study comprised two phases: a) selection and structuring of criteria through a targeted literature review and scientific committee (SC) consensus; and b) nonhierarchical weighting of criteria and sub-criteria by a multidisciplinary expert group (n = 19) of hospital pharmacists, physicians, and evaluators/decision-makers.
RESULTS: Twelve criteria were identified. Following SC discussion, five criteria were selected for weighting: efficacy, safety, patient-reported outcomes, treatment cost, and other direct medical costs. Among the weighted sub-criteria, overall survival (OS) and progression-free survival (PFS) achieved the highest scores, with mean values of 4.21 (95% CI: 3.68-4.74) and 4.05 (95% CI: 3.52-4.58), respectively.
CONCLUSIONS: In this structured MCDA-informed expert weighting exercise, OS and PFS were identified as the highest-priority value criteria for designing and monitoring outcomes-based MEAs for CAR-T therapies in RRMM. These findings may support feasible, evidence-based reimbursement models but require validation in larger and more diverse stakeholder groups.